Exon skipping and inclusion therapies methods and protocols - Yokota, Toshifumi ca. 20./21. Jh; Maruyama, Rika ca. 20./21. Jh (Springer New York, 2018)
Invention and Early History of Exon Skipping and Splice Modulation – An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases – Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy – Nusinersen in the Treatment of Spinal Muscular Atrophy – Tips to Design Effective Splice-switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion – Antisense Oligonucleotide Targeting of 3'UTR of mRNA for Expression Knockdown – Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro – Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-m Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression – In vitro Multi-exon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient –